Top News

Illustration of mitochondria linked to rare disease drug research and biotech innovation

RESEARCH

20 Sep 2025

From Niche to Notable: Rare Diseases Reshape Biotech

Approval of elamipretide and Mirum Pharma deal point to growing investor interest in high-need therapies with small patient populations

Newborn baby with hearing test and forehead monitoring equipment in a clinical setting

TECHNOLOGY

13 May 2026

Born Deaf No More: FDA Greenlights Historic Treatment

BridgeBio Attruby medicine box for transthyretin amyloid cardiomyopathy

INSIGHTS

30 Oct 2025

BridgeBio’s Strategy Shift Tests the Economics of Rare Disease

Latest News

One Drug, Five Diseases, Zero Approved Cures Until Now

Hospital dialysis room with a dialysis machine next to a patient bed and large Aboriginal artworks on the wall

INNOVATION

3 Jun 2026

Travere licenses civorebrutinib from Everest in $1.14B deal targeting rare immune-mediated kidney diseases

RNA strand illustration representing personalised antisense therapy development

INNOVATION

1 Feb 2025

Inside the Push to Personalize RNA Medicine

Tailored antisense drugs move from concept to clinic as researchers and companies test faster models for ultra-rare conditions

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